By Andrew M. Seaman
NEW YORK (Reuters Health) - Research trials that evaluate drugs and other treatments for children don't always align with needs around the world, according to a new analysis.
Researchers compared trials registered on a U.S. government website to the burden of 21 diseases found in countries around the world. They found that what was being researched was only moderately tied to what trials are needed.
"In pediatrics in general, there is a paucity of the information available to us on how to use drugs and interventions," Dr. Florence Bourgeois told Reuters Health.
Bourgeois is the study's lead author and an assistant professor at Harvard Medical School in Boston.
"We decided to look at if the resources we do have available and the trials that are being conducted are addressing the areas of greatest needs," she said.
Historically, children have been underrepresented in trials that evaluate the safety and effectiveness of drugs (see Reuters Health article of Jul. 24, 2012 here: http://reut.rs/18UoeRM.
Dr. Kenneth Mandl, the study's senior author, told Reuters Health that not evaluating drugs in children leads doctors to prescribe medicine "off label," which means it's ordered to treat someone or something other than its approved use.
"It's very important to look at the distribution of what is being done versus the needs," Mandl, a professor at Harvard Medical School, said.
For the new study, the researchers compared all studies that evaluated a drug or treatment in people younger than 18 years old and were started after 2006 and registered on ClinicalTrials.gov by May 2, 2012.
The trials were separated by subject area - such as injuries or infectious diseases. They were then compared to global rates of those diseases as measured by the World Health Organization (WHO).
Overall, the researchers compared 5,373 trials registered on the website to global rates of 21 different conditions. They found that the registered trials moderately matched the global disease burden.
For example, based on how many kids are affected by neuropsychiatric disease - such as mood and behavior disorders, the researchers estimated an excess of 524 trials for that condition.
On the other hand, injuries were the most understudied condition. The researchers estimated there was a deficit of 260 trials for that category.
For some conditions like genitourinary disease, which includes kidney failure, the registered trials matched the global disease burden.
When the researchers divided countries into high-, middle- and low-income categories, the imbalances between disease burden and expected number of trials varied, with the greatest disconnect seen in poor countries.
In addition, despite 79 percent of the research occurring in high-income countries overall, those countries only made up about 2 percent of the global disease burden, the authors write in the journal Pediatrics.
Meanwhile, low-income countries represented 73 percent of the disease burden, but only 7 percent of the research took place there.
"We have this laissez-faire attitude of how health research will precede - that companies, government and foundations will identify the most important areas to do trial and analyses," Dr. Clay Johnston said. "This type of research shows that doesn't happen as well as we'd like."
Johnston is the associate vice chancellor of research at the University of California, San Francisco. He was not involved in the new study, but previously examined research funding compared to global disease burden.
He told Reuters Health that research is typically driven by industry and the overrepresentation and underrepresentation of trials for certain conditions may represent where there are markets for drugs.
For example, the new study showed a surplus of trials for neuropsychiatric conditions, which may be treated by a multitude of different medications. The deficit of trials for poor nutrition may represent an area where there is no market for those companies.
"Those are areas that tend to be neglected unless they're picked up by organizations," Johnston said. He added that the new report may be useful to nonprofit organizations that conduct research in those neglected areas.
"It empowers them and reminds the rest of us how important they are and how useful it is for them to work in these areas," he said.
Bourgeois said it's possible to monitor these estimates over time with the data from ClinicalTrials.gov.
"We really need to focus on using our resources most effectively and where there is room for improvement," she said.
But Johnston said it's difficult to simply shift funding toward neglected areas, because there is also promising and needed research being conducted in overrepresented areas.
He said the new study is more of a report card that shows interesting information "to take a step back and ask globally, ‘how are we doing?'"
SOURCE: http://bit.ly/1elR6XG Pediatrics, online December 16, 2013.